BioMarin Submits Vimizim MAA To EMA For The Treatment Of MPS IVA

SAN RAFAEL, Calif., April 24, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. A Biologics License Application (BLA) for Vimizim was submitted to the U.S. Food and Drug Administration (FDA) in March 2013.

"The submission of this application to the EMA represents another notable milestone in our efforts to bring the first therapeutic option to patients with MPS IVA worldwide," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "MPS IVA is a serious debilitating disease with no treatment options. We hope to leverage our expertise in developing enzyme replacement therapies to introduce a life-altering therapy and change the course of the disease." 

Mr. Bienaimé continued, "Earlier this year, the EMA accepted our request for accelerated assessment for this MAA based on the premise that Vimizim could satisfy an unmet medical need and is of major interest from the point of view of therapeutic innovation and public health. We look forward to working with the European regulatory authorities in the coming months to bring this therapy to patients in need."


Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disease characterized by deficient activity of N-acetylgalactosamine-6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.

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